• Why investment in early stage drugs must continue for a healthy biotech industry
• Can investors in biotech achieve their ROI in the expected timeframe?

• The EMEA roadmap
• EU research programmes
• New cell and tissue legislation
• How biosimilars are being dealt with in Europe

• Public: Private Partnerships
• Fund raising as a service business
• National Biomanufacturing Centre

• How to prepare for unexpected turbulence,
• What key decisions you will need to make along the way
• How to avoid treacherous pitfalls
• Problems for European biotechs

• Outsourcing vs in-house production
• The Intercell model
• Why Scotland?

• Why we floated
• What had to be done to get us ready for IPO
• What happened
• Lessons learned

• How to make biologicals as a small biotech
• Buying vs making
• Key selling points when licensing out
• Adapting you manufacturing strategy as the company grows
• The Celltech model - could it work for you?

• Use of different techniques in licensing deals and acquisitions
• Portfolio approach for the asssesment of L&A projects versus internal R&D projects
• Why current valuation tools do not always allow us to assess the 'go/no go' decision robustly
• Real option valuation to bridge the gap between 'conventional' valuation tools and strategic valuations

• Alliance/license strategies
• Issues determining an alliance structure
• Case study 1: contractual framework to include licenses, substance bulk supply, development services, etc
• Case study 2: co-venture agreement: co-promotion countries, co- marketing countries, single presence countries

• The interface between big pharma’s business development and biotech process development
• How to create a partnership where there is an acceptable valuation in terms of NPV
• Criteria for in-licensing and how it is changing
• What kind of results does big pharma look for term of pre-clinical tests?

• Technical feasibility
• Regulatory compliance
• Key elements in the equation of clinical trial material supply
• Economic viability – can you extrapolate during early product development?

• New technologies offer improvements in costs and performance
• Benefits come at some risk of the unknown
• Procedures for evaluating and integrating new technologies to minimise risks to timelines
• Impact is greatest upstream, but improvements downstream are also required

• The scientific and regulatory issues that led to the adoption of the principles described in ICH S6
• Unique issues that must be considered in the design of biopharmaceutical development programmes
• Key considerations for the design of efficient strategies

• Detailed physicochemical and biological characterisation early in product development for better understanding of the molecule (consistent with ICH Q6B)
• Identifying post-translational modifications to assist in developing an optimal process and relevant stability assays
• Early characterisation of product stability profile to assist with decision-making for candidate molecule
• Defining the site of structural changes for assessing whether the change is likely to have an impact on potency or safety

• Definition of the company´s product development strategy with process in mind
• Requirements for early clinical trial material balanced with new process options
• Which investigations (CMC and non-clinical) are mandatory and which are optional?
• What is the real challenge between cost and quality during product development?

• High throughput automated microbioreactors
• Process optimisation
• Maximising return form process development resources
• Minimising time from clone to manufacturing and the clinic

• Degrees of virtuality – the benefits and the drawbacks
• What to outsource and what to keep in-house
• How to select a contractor
• Getting value for money – does low cost always mean high risk?
• Managing the process

• Defining the technology that fits your needs
• Choosing established or entrepreneurial technology
• Establishing the right commitment for contract activities
• Achieving success with concurrent development support

• Who has the best regulatory expertise?
• Options for choosing cell line development for high yields
• The need to assess process development expertise, costs and service
• A discussion of the national centers and how they serve the SMEs

• China and India emerging as major forces on the pharma outsourcing space
• Their traditional sales proposition revolving around low delivered prices
• The increasing number of pharmaceutical companies considering these countries as possible sources
• Assessing the risks associated with these countries against ultimate rewards achievable

• The limitations of available assays for predicting immunogenicity of biologicals
• Available assays for measuring antibodies in patient sera
• Assays for characterising immune responses as part of unwanted immunogenicity assessment
• Animal models

• Factors contributing to the immunogenicity of therapeutic proteins
• Potential effects when a therapeutic protein is immunogenic
• Clinical example of immunogenicity

• Examples of comparability
• Comparability intra and inter companies
• Impact of protein complexity

• A very young industry presenting difficulties for regulators
• Do generic firms focus too much on manufacturing rather than regulatory issues?
• The approval process as the key issue
• The likelihood of antibodies and more complex proteins being copied
• When will there be the first biogeneric launches?

• The guideline on comparability of medicinal products containing biotechnology-derived proteins
• Impact of Clinical Trials Directive 2001/20/EC
• Legal issues
• Framework for approval and policy issues

• US-EU statutory recognition that biological medicines present special issues
• US regulatory framework and debate about “follow-on biologics” and “505(b)(2)”
• EU regulatory framework and its evolution: ambiguities and issues
• U.S.-EU quandary: about what data will be required?
• U.S.-EU issue: may a regulatory reviewer rely on a reference product’s data in reviewing biosimilar application?
• The innovator’s trade secrets?

• Pre-clinical and clinical progress to date
• How to transform the clinical development of stem cells into viable pharmaceutical products
• Latest advances in human stem cells, cord blood and bone marrow stem cells and expansion
• Scientific and legal issues surrounding embryonic stem cell research
• Expectations for future possible manufacturing of such products

• Technology
• Clinical and regulatory aspects

• Design and design development
• Training and process familiarisation
• Organisational issues
• Tech transfer agreement and tech transfer plans
• Execution and data generation
• Filing strategies and project management

• Decreasing experimental duration (= throughput) by PAT and PIMS
• Increasing data reliability and reproducibility (= data quality) by PAT and PIMS
• Monitoring of biological process state variables and data density determine data quality
• Future application of PAT and PIMS for “parametric release” in biopharmaceuticals production

The trend towards disposable processing systems in biopharmaceutical manufacturing continues, driven by the considerable economic and operational advantages of these systems.  This presentation describes a single-use stirred-tank bioreactor system that has been developed by HyClone Laboratories, Inc., and Baxter Healthcare Corp.

• The implementation of Directive 2001/20/EC into EU Member State legislation
• The development of ethical review and an ethical framework for clinical research in Europe
• The impact of the Directive on the sponsoring and management of clinical trials
• The relationship between the Directive and clinical trials registries

• Critical changes in the process from a European and global perspective
• Management of the transition period: national rules interpretation
• Redesign of the Phase IV strategy in Europe
• Multi-country, multi-center trials for rare diseases and cancer
• Lessons learned and areas for future clarification

• Achieving quality criteria to enter clinical studies
• Achieving GMP requirements in early stage development
• Turning a hurdle into a benefit for product value
• Applying scientific advice
• Strategic approaches to the Clinical Trial Directive

• Background to the implementation of The EU Directive
• The Directive and Good Clinical Practice
• The new clinical research environment created by the Directive
• Implications for clinical research
• Is there a problem with inconsistent implementation of the Directive across Europe?

• Key learnings from common audit findings
• Building the function and use of vendors for internal and external quality management
• Common misunderstandings in clinical quality management
• Identifying the added value of quality management

• Examples of manufacturing changes
• The new guideline from CPMP

• Strategies for comparability exercises
• A review of FDA and ICH guidelines for assessing product comparability: including characterisation, specifications and analytical methods
• Discussion of the ICH Q5S guideline
• How biosimilar products are influencing definitions of comparability

• Designing appropriate product characterisation studies
• Analytical, pre-clinical and clinical where necessary
• The need for process validation and process comparability
• Balancing resources by identifying critical parameters

• Planning comparability: the easy part - why, what, how?
• Regulatory guidelines, analytical methods, the protocol
• Demonstrating comparability - the tricky part
• Case study – process changes during monoclonal antibody production.
• Conclusions

• Is it a useful approach to defer development cost?
• Minimising the technical risk post-approval
• What is the experience within in the industry?
• What is the experience within the regulatory authorities?

The trend towards disposable processing systems in biopharmaceutical manufacturing continues, driven by the considerable economic and operational advantages of these systems.  This presentation describes a single-use stirred-tank bioreactor system that has been developed by HyClone Laboratories, Inc., and Baxter Healthcare Corp.

• Exploitation of the cell's secretory pathway through modified mRNA targeting
• Identification of specific targeting signals
• Up to 50 fold increased yields for naturally secreted proteins
• Efficient secretion of naturally intracellular proteins
• “Proof of concept" provided with various biomedically important proteins

• The egg as a bioreactor
• Characterisation of recombinant proteins produced in the egg white
• The importance of developing an alternative avian transgenic platform
• Production of transchromosomic chickens

• Critical parameters related with the scale-up and the commercial manufacturing of biopharmaceuticals
• The biochemical properties of the product to be manufactured
• How assembly and post-translational modifications will affect decisions
• Production volume and costs for each system
• How to combat the regulatory issues associated with scale-up
  

• The outline of FDA’s and ISPE’s initiatives
• Increased focus on product and process understanding
• Using PAT as the driver to implement change
• Lean manufacturing and lean GMP
• Fast track engineering based on modular process concepts 
• Upgrading your facilities in record time

• Strategies for accelerated start-up and site approval
• Risk-based approach for process validation, with focus on upstream processing
• Process transfer from another site and comparability concept
• Rationale to establish comparable process and product

• Solvay’s pioneering efforts for flu vaccine manufacturing
• Outline of Stedim’s disposable process initiatives, with particular emphasis on RAFT (Rapid Aseptic Fluid Transfer system)
• Explanation of how Solvay works with the RAFT system in final formulation & filling
• What lead to Solvay’s choice for installing the RAFT system?
• Installation, validation and regulatory approval of the system
• Economic model comparing the RAFT system with traditional hard piped systems

• Technologies and platforms in development
• Cost contributions and cost optimisation
• Constraints and restrictions in downstream processing
• A look at the future

• Process development with full pipelines and empty pockets
• Biomanufacturing: the current backlog in bioseparation
• Orthogonal biosafety concepts
• Disposable manufacturing and process economy

• Handling large volume of dilute product in a speedy manner

• Looking beyond Protein A affinity chromatography for antibody purification
• Review of alternate methods and sorbents
• Application of mixed-mode ligands for capture of antibodies and other proteins
• Miniaturised “on-chip” chromatography process development approach

• What does higher selectivity mean for downstream processing?
• Technologies for discovering process-suitable ligands
• Issues around media cost, column lifetime and ligand leaching
• Applications of diversity technologies